‘Three more months is a lot if you have six months to live’ (Guest blog)
29.05.19
Bettina Ryll, a medically-trained scientist, started the Melanoma Patient Network Europe after her husband died of the disease. She knows the value of extending survival and improving quality of life.
‘Unless you have experienced a terminal cancer diagnosis, you have no concept of what three more months would mean at the end of your life,’ said Dr Ryll. ‘When you’re not sick, three months seems like nothing. When you know your time is limited, three months can mean a lot, especially when the quality of life is good.’
Dr Ryll was asked for her view of a WHO technical report on the pricing of cancer medicines which was critical of the cost of drugs that add around three months to patients’ lives. It says that while therapies approved in the past 15 to 20 years improve survival, ‘the size of the benefit may still be small; the average benefit is 3 months, which may be considered marginal by clinical experts.’
Dr Ryll’s family lived the reality of incurable cancer when her husband was diagnosed with metastatic melanoma in 2011. His life expectancy at that time was between six and nine months.
‘I don’t think it’s helpful to integrate across 15-to-20 years of data from multiple types of drugs and several forms of cancer to start with,’ she said. ‘Cancer isn’t one disease, prognoses and outcomes vary greatly, so something like three months will mean something very different depending on the context. And as we have learned from immune therapies in melanoma, median survival can be misleading when considered without the spread of the data: many get no or very limited benefit but some actually survive a disease that was previously invariably deadly.’
Quality counts
However, quantity is not everything. It is the quality of the prolonged life that matters to patients. Dr Ryll is somewhat indignant: ‘Everybody thinks of cancer therapy as chemotherapy with serious side effects; some of them visible like hair loss,’ she said. ‘Something that causes great upset among patients with metastatic melanoma is that they are often told they can’t be suffering from cancer because they look so well!’
‘Chemotherapy never worked in melanoma and the therapies we got now are from very different therapeutic classes – they look nothing like chemotherapy. On targeted therapy, my husband certainly had side effects like a rash and dry skin but the fact that the huge tumour that has become so painful, disappeared, meant that his quality of life dramatically improved – he regained the use of his arm.
‘Even if time-wise it wasn’t as much as we had hoped for, that alone was hugely valuable to us. He actually had no side effects on immune therapy, but we have other patients who suffer greatly, so this is one of those therapies where side effects are highly unpredictable.’
Dr Ryll’s family had some frank discussions during her husband’s illness. ‘Suffering is what people fear,’ she said. ‘If a new drug can reduce suffering, even if it doesn’t prolong life, society should value this.’
Even a little bit of extra time can make a big difference to how the family deals with their loss afterwards. ‘It gives you time to prepare. We got some breathing space where he, for a while, hardly needed any painkillers. It was the calm before the storm which we knew would come – but that’s when we talked about things like his wishes for his daughters’ future. In terms of dignity and humanity and in meaning for our family, these things are hard to capture in sheer numbers when we ask how much a treatment is worth.’
Patient advocacy
Dr Ryll founded the Melanoma Patient Network Europe and patient-centric drug development is her passion. She is a deeply knowledgeable patient advocate, engaging with other healthcare players on the nuts and bolts on how drugs are developed, approved and reimbursed. Combining personal experience with medical training and practical research experience, she is tirelessly searching for constructive solutions that fulfil every stakeholder’s needs without ever letting them forget that it is the patients who pay the ultimate price.
Dr Ryll said there is a real tension between affordable access to medicines and the need to invest in innovations that address unmet need. ‘The current price level of some new medicines is de facto limiting access to that very innovation,’ she said. ‘Whether we would have had that innovation without the incentives remains open for discussion. The reality for us is that melanoma patients without access to the new drugs still die just as they did in the days when my husband was diagnosed.’
For clinicians and decision-makers, this tension may be a factor for some of the criticisms of new medicines. ‘There is an element of sour grapes to some of the arguments – and it’s not very constructive,’ she said. ‘The attitude is that because current prices are an issue, we should become more critical of the drugs themselves. The debate has become more heated.’
Wanted: New approaches
It is time for a different approach, she said, acknowledging that the problem is not easy to solve. ‘These are really two interrelated problems: on the one hand, there are conditions for which there are no treatments and people are dying. On the other, there are new treatments but some people cannot access them due to price and are dying yet again,’ she explained. ‘The problem is that our current way of trying to solve one of the problems aggravates the respective other’.
Dr Ryll advocates medical innovation that improves outcomes that matter to patients, but says new medicines that sit on the shelf are of no value: ‘That’s not innovation it’s just an invention. We need to incorporate access into our concept of innovation.’
Despite the challenges, she is overall rather optimistic. ‘Pressure tends to be a prelude to change, and there is a growing appreciation that we need to strike a better balance between incentives for innovation and access.’ Dr Ryll favours pragmatic solutions – such a risk-sharing agreements and conditional reimbursement schemes – that grant patients in desperate need access to new drugs while systematically gathering evidence on how that new product behaves in the real world.
‘Letting loose an unproven treatment on a vulnerable patient population without safeguards is as irresponsible as letting desperate patients ‘die in safety’ until the last uncertainties are solved,’ she says. ‘If the alternative is certain death, some patients will accept considerable risk – this does not release society from its responsibility to protect these patients. On the other side, concerns about uncertainty at regulatory and financial level are absolutely valid but should not serve as an excuse to let people die because if there is one many patients don’t have, it is time.’
